Gene Therapy for CF using Canonic Liposome Mediated Gene Transfer: Phase 1 Trial 
INFORMED CONSENT 
I have been asked to take part in a Research Study. Before agreeing to participate, it is 
important that I read and understand the following explanation. I understand that I will be one 
of about nine (9) subjects in this study. 
PURPOSE 
Human genes carry functions which are necessary for healthy human life. Cystic fibrosis 
(CF) is caused when one of these genes, called the cystic fibrosis gene, cannot function 
properly. As a result of this dysfunction, tissues in the bodies of CF patients (and the cells which 
are the building blocks of these tissues) cannot move salts properly. For example, the high 
content of salt in the sweat of CF patients, and the thick respiratory mucous in the lungs of CF 
patients, have both been associated with difficulties moving salts such as sodium and chloride. 
The purpose of this study is to determine whether normal, functional copies of the CF 
gene can be delivered to the cells lining the nose of CF patients. This study will use small 
amounts of molecules of fat (also called "lipids") to carry the normal gene into these cells. 
This study has two goals. The first goal is to determine whether lipids can be used to 
safely carry the normal gene into the cells lining the nose. The second goal is to determine 
whether after receiving the normal CF gene, the inside of the nose will develop an improved 
ability to move salts. 
The use of genes to treat human diseases has been called "gene therapy". Because the 
serious complications of CF occur in parts of the body other than the nose (for example, in the 
lungs and pancreas), it is unlikely that the delivery of a normal CF gene to the inside of the nose 
will make participants in this study healthier. On the other hand, the overall goal of this research 
study will be to allow the possibility of attempting the same type of normal gene delivery to CF 
lungs, with the hope of developing better treatments for cystic fibrosis. 
DURATION 
I understand that I will participate in this study for approximately five (5) months. I will 
be evaluated in the Cystic Fibrosis Clinic at The Children’s Hospital of Alabama at 28 days, 14 
days and 1 day prior to receiving the CF gene. Each of these evaluations will take approximately 
three (3) hours. On the day the gene is given, I will be admitted to the Clinical Research Center 
at The Children’s Hospital of Alabama and will remain in the hospital for a period of 
approximately five (5) days. 
After this period, if there are no other significant medical problems, I will be discharged 
and followed on a daily basis in the Outpatient Clinic for one week (7 days), then twice a week 
for the following three weeks, and then every 2-3 weeks for the next 10 weeks. After this 
period, I will keep my regular Cystic Fibrosis Clinic appointments and be seen approximately 
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Recombinant DNA Research, Volume 18 
